How one man’s lack of vision inspired healios to blaze a trail for hiPS Ccell therapies
The first afternoon session of WSCRM 2014, focusing on industrialisation of iPSC cells, saw Dr Hardy TS Kagimoto tell the story of Healios’ iPSC-derived therapy for age-related macular degeneration (AMD) to human trials, the first of its kind globally.
Dr Kagimoto spoke of the difficulties in obtaining funding from venture capitalists owing to the unique nature of his team’s project, ‘no one knows what the timeline for this project will be’, he offered candidly, no one had attempted anything of its type in earnest before Healios’ efforts. In consensus with views expressed by speakers, such as Dr Greg Bonfiglio and Dr Michael West, in the morning session, the Healios president admitted that finding investors willing to view his long-term project as a viable investment.
The Healios President and CEO went on to describe the astounding progress his company have made with collaborators, Shibuya Kogyo Co. Ltd., in developing a regulatory approved GMP production process for iPS-derived retinal pigment epithelium cells. Healios estimate they are able to produce 30,000 therapeutic units, suitable for transplantation, every year using a specially designed automated platform; enough to satisfy annual demands for such a therapy in Japan. This is a mind-blowing achievement considering the widely held view that a key challenge faced by companies operating in this area is developing cost-effective, GMP-based, robust scale up strategies that are capable of satisfying market demands in terms of annual doses required.
It is testament to the philosophy of Healios and its owners that this project is going forwards with no economic incentives attached to its duration; attention is directed solely towards the development of a safe, efficacious AMD therapy. Keen to avoid a repeat of the safety controversies that afflicted early gene therapy trials carried out by Gelsinger, Dr Kagimoto stressed that the first human trials would have a primary focus on the safety of his company’s therapy before moving forward to prove its in vivo efficacy in humans. Indeed, it is the rigorous attention to safety in pre-clinical studies that persuaded regulatory authorities in Japan to green light human trials in the first instance.
Speaking with an infectious enthusiasm for his project, Dr Kagimoto exhibited the drive and determination necessary to see his vision of a cure for age-related macular regeneration realised.
Michael Jenkins for Total BioPharma at WSCRM Congress, London
Michael is an EngD research student at University College London. His work, in collaboration with Neusentis Ltd., investigates cost of goods modelling and economic optimisation of hiPSC bioprocesses.